Rare Daily Staff
Danish dermatology specialists LEO Pharma and California rare disease drug developer PellePharm said they have entered a strategic development and commercialization collaboration to develop therapies for rare skin diseases.
Under the terms of the agreement, LEO Pharma has initially committed $70 million in equity financing and financial R&D support to fund the global phase 3 trial for patidegib topical gel 2 percent for the prevention and treatment of Gorlin Syndrome. LEO Pharma has an option to acquire all shares in PellePharm. PellePharm and its stockholders could receive up to an additional $690 million including merger consideration, and regulatory and commercial milestone payments. In addition, PellePharm stockholders are eligible to receive a double-digit royalty after achieving certain commercial milestones.
Gorlin syndrome is a rare, genetic disease characterized by heritable mutations to a tumor suppressor gene, which acts as the primary inhibitor of the hedgehog signaling pathway. This leads to the formation of multiple basal cell carcinomas, often on the face. There are no approved treatments for Gorlin syndrome.
Patidegib topical gel has shown early promise in a phase 2 clinical study for the mitigation of BCC tumors in Gorlin syndrome by blocking the disease at its source within the hedgehog signaling pathway. The topical formulation of patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in phase 1 trials without the adverse systemic side effects.
The U.S. Food and Drug Administration has granted PellePharm Orphan Drug designation and Breakthrough Therapy designation for patidegib topical gel in Gorlin Syndrome.
“Our company is founded on the commitment to targeting rare dermatologic diseases at the source and bringing new groundbreaking treatments forward to patients as efficiently and effectively as possible,” said Sanuj Ravindran, president and CEO of PellePharm. “As a global leader in medical dermatology, LEO Pharma is a great fit as both a development and commercialization partner. This collaboration puts us on track to commence our pivotal phase 3 Gorlin Syndrome trial in early 2019.”
November 20, 2018
Photo: Sanuj Ravindran, president and CEO of PellePharm.